Objective To explore the mechanism of Yisuishengxue Formula in the treatment of thalassemia. Methods The changes of hemoglobin (Hb), red blood cell count (RBC), reticulocyte count (Ret) and fetal hemoglobin (HbF) were observed before and after treatment in 30 children patients treated with Yisuishengxue Formula. The influences of Yisuishengxue Formula on the gene expressions of transcriptions, including GATA-binding protein-1 (GATA-1), GATA-2, erythroid Krüppel-like factor (EKLF), Krtüppel-like transcription factor (FKLF) and nuclear factor erythriod 2 (NF-E2) in karyocytes were detected in 10 children patients by using real-time fluorescence quantitative polymerase chain reaction (RT-PCR). Results After the treatment, all hematologic parameters were improved in the patients and were significant (P ＜0.01) compared with those before the treatment. The gene expressions of GATA-1, GATA-2 and NF-E2 increased and were significant compared with those before the treatment (P ＜ 0.01, P- ＜ 0.05). The differences in gene expressions of EKLF and FKLF were no significant before and after the treatment. Conclusion Yisuishengxue Formula can relieve the symptoms of the patients with thalassemia. It controls the expression of globin gene cluster and improves the generation of effective erythrocytes through regulating the expressions of erythroid specific trans-acting factors, which is one of molecular mechanisms of its therapeutic effect.%目的 探讨益髓生血方治疗珠蛋白生成障碍性贫血的作用机制.方法 观察30例患儿用益肾活血方(山萸肉、熟地黄、补骨脂、何首乌、阿胶、鳖甲、黄芪等)治疗前后血红蛋白(Hb)、红细胞(RBC)、网织红细胞计数(Ret)、胎儿血红蛋白(HbF)的改变;通过实时荧光定量PCR检测该药对10例患者骨髓有核细胞中转录因子GATA模序结合蛋白(GATA-1和GATA-2)、红细胞Krüppel样因子(EKLF)、与EKLF结构相近的红系特异转录因子(FKLF)和红细胞核因子(NF-E2)基因表达的影响.结果 治疗后患者各项血液学参数均有提高,与治疗前比较差异有显著性(P＜0.01);骨髓有核细胞GATA-1、GATA-2和NF-E2基因表达明显升高,与治疗前比较差异有显著性(P＜0.01、P＜0.05);治疗前后EKLF、FKLF基因表达没有明显差异.结论 益髓生血方可以改善患者的临床症状,通过调节红系特异反式作用因子基因的表达、调控珠蛋白基因簇的表达、促进有效红细胞生成而达到治疗该病的作用,这也是益髓生血方作用的分子机制之一.
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